Journal of thrombosis and haemostasis : JTH
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J. Thromb. Haemost. · Nov 2020
Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.
Gene therapy for people with hemophilia (PWH) will soon become available outside current clinical trials. The World Federation of Hemophilia (WFH), in collaboration with International Society of Thrombosis and Hemostasis Scientific and Standardization Committee (ISTH SSC), the European Haemophilia Consortium (EHC), the US National Hemophilia Foundation (NHF), the American Thrombosis and Hemostasis Network (ATHN), industry gene therapy development partners and Regulatory liaisons have developed the Gene Therapy Registry (GTR), designed to collect long-term data on all PWH who receive hemophilia gene therapy. ⋯ The GTR is a global effort to ensure that long term safety and efficacy outcomes are recorded and analysed and rare adverse events, in a small patient population, are identified. Many unknowns on the long-term safety and efficacy of gene therapy for hemophilia may also be addressed.
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J. Thromb. Haemost. · Oct 2020
Circulating endothelial progenitors are increased in COVID-19 patients and correlate with SARS-CoV-2 RNA in severe cases.
During the course of COVID-19, the disease caused by the new coronavirus severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), thrombotic phenomena and/or diffuse vascular damage are frequent, and viral elements have been observed within endothelial cells. ⋯ CD146 + CECs and CEPs might be investigated as candidate biomarkers of endothelial damage in COVID-19 patients.
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J. Thromb. Haemost. · Oct 2020
Evaluation of oral factor Xa inhibitor-associated extracranial bleeding reversal with andexanet alfa.
A reversal agent for factor Xa (FXa) inhibitors, andexanet alfa, was Food and Drug Administration approved without extensive study of clinical effectiveness, due to an overwhelming demand for FXa inhibitor reversal. In this study, we aimed to describe patient selection, clinical effectiveness, and safety of FXa inhibitor reversal with andexanet alfa in patients presenting with extracranial bleeding. ⋯ We report poor overall outcomes, a low rate of hemostatic effectiveness, and a high rate of ischemic complications and mortality in this retrospective analysis of oral FXa inhibitor reversal with andexanet alfa for extracranial bleeds. More rigorous epidemiological, and ideally randomized studies, are needed to determine the role of andexanet alfa for FXa inhibitor-associated bleeding for extracranial hemorrhages, where large variation in severity and presentation exists.
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J. Thromb. Haemost. · Oct 2020
Chronic therapeutic anticoagulation is associated with decreased thrombotic complications in SARS-CoV-2 infection.
Thrombotic disease complicates severe SARS-CoV-2 infection and is associated with increased morbidity and mortality. Various anticoagulation strategies have been evaluated in hospitalized patients to prevent complications. The impact of chronic anticoagulation before SARS-CoV-2 infection on the risk for subsequent thrombosis has not been systematically studied. ⋯ Our uncontrolled findings suggest that chronic anticoagulation at the time of infection may protect against thrombotic complications and decrease disease severity.